Acute Myeloid Leukemia: A Triplet of Oral Drugs for the Most Fragile

Improving treatment for acute myeloid leukemia, especially for the most fragile patients who cannot receive chemotherapy. This is the goal of a clinical study (phase 1-2) presented at the European Congress of Hematology (Eha) held in Milan. The trial tested the addition of the drug venetoclax to the fixed-dose combination of decitabine-cedazuridine (already approved in Italy by AIFA) in newly diagnosed patients aged 75 years or older, and not eligible for standard intensive chemotherapy.

Who does acute myeloid leukemia affect?

Acute myeloid leukemia is a rare blood cancer, but it is also the most common form of acute leukemia, which in Italy accounts for 3,500-4,000 cases per year, especially among men and especially between the ages of 55 and 60. The incidence throughout Europe is also increasing, due to the aging of the population.

An oral formulation

The decitabine/cedazuridine formulation was developed specifically for older patients, which allows for daily oral administration of decitabine for five days (from day 1 to day 5 of each 28-day cycle): in fact, cedazuridine guarantees systemic exposure of decitabine comparable to that of intravenous decitabine, thus simplifying the administration and management of the therapy. However, there are still unmet needs, as Adriano Venditti , Full Professor of Hematology at the University of Rome Tor Vergata, points out. “The oral combination of decitabine/cedazuridine and venetoclax has provided very promising preliminary results and could represent an important therapeutic option in the future,” he explains. “The future possibility of having a completely oral therapy would represent a decisive change both in terms of impact on the quality of life of these patients and in terms of management of the care and hospital burden.”

The study data

Study results show that 46% of patients achieved complete remission of the disease: 80% maintained it at 6 months and 75% at 12 months. Median overall survival was 15.5 months. Almost all participants (98%) reported treatment-related adverse events of grade 3 or higher, most commonly febrile neutropenia (49.5%), anemia (38.6%), and neutropenia (35.6%). The 30- and 60-day mortality rates were 3% and 9.9%, respectively.

Towards a new possibility of treatment

“The decitabine/cedazuridine combination with venetoclax could represent a new therapeutic opportunity in the current landscape, with a significant impact in reducing the hospital load, but above all in improving the quality of life of patients, their families and caregivers - concludes Alessandro Lattuada, Managing Director of Otsuka Italia - We hope for the approval of the therapy by the European regulatory body as soon as possible to allow rapid access to European and Italian patients”.